Wave Life Sciences Highlights Recent Achievements and Upcoming 2024 Milestones
08 Januar 2024 - 2:00PM
Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage
biotechnology company focused on unlocking the broad potential of
RNA medicines to transform human health, today announced key 2024
milestones across its clinical programs, growing pipeline, and
leading RNA medicines platform.
“Wave is uniquely positioned to build the world’s leading RNA
medicines company, with a clinically validated platform,
diversified pipeline aimed at high-impact diseases, and meaningful
commercial opportunities. Powered by multiple RNA modalities, a
decade of chemistry innovation, and deep genetic insights, we are
opening up new areas of disease biology and designing optimal ways
of treating rare and common diseases. We expect 2024 will be an
inflection year that will drive significant value for Wave, our
shareholders, and most importantly, for the patients who will
benefit from our research,” said Paul Bolno, MD, MBA, President and
Chief Executive Officer of Wave Life Sciences.
Dr. Bolno continued: “This year, we expect the first-ever
clinical proof-of-mechanism data for RNA editing with WVE-006 in
AATD, which will accelerate additional Wave RNA editing programs.
We are rapidly advancing our wholly owned INHBE program for
obesity, which is designed to enable healthy, sustainable fat loss
and address limitations of GLP1s. We also expect to deliver data
demonstrating production of endogenous dystrophin protein in DMD
with WVE-N531, as well as multidose data for WVE-003, our HD
candidate that is uniquely designed to lower mutant huntingtin and
maintain healthy, wild-type huntingtin. These programs underscore
our drive to ‘Reimagine Possible’ for science, for medicine, and
for human health.”
2024 Priorities and Anticipated Milestones
Advance AATD program to clinical proof-of-mechanism data
and unlock additional opportunities for Wave’s leading RNA editing
capability
WVE-006 in Alpha-1 Antitrypsin Deficiency (AATD) & RNA
Editing
- WVE-006 is a GalNAc-conjugated, subcutaneously administered RNA
editing oligonucleotide and is the first-ever RNA editing candidate
to be evaluated in humans. WVE-006 is uniquely designed to correct
the disease-causing SERPINA1 Z transcript mutation in AATD, thereby
restoring circulation of wild-type M-AAT protein and reducing Z-AAT
protein levels to address both lung and liver manifestations of the
disease.
- In preclinical studies, WVE-006 demonstrated potent and durable
editing of SERPINA1 Z transcript in mice, restoration of AAT
protein up to 30 micromolar, and improvement in several markers of
liver disease. WVE-006 is also highly specific with no evidence of
bystander editing.
- In December 2023, Wave initiated dosing in healthy volunteers
in the RestorAATion clinical trial program investigating WVE-006 as
a potential treatment for AATD. RestorAATion includes healthy
volunteers (RestorAATion-1) as well as individuals with AATD who
have the homozygous PiZZ mutation (RestorAATion-2) and is designed
to provide an efficient path to proof-of-mechanism as measured by
restoration of M-AAT protein in serum.
- GSK has an exclusive global license to WVE-006 as part of its
strategic collaboration with Wave. With initiation of dosing in
RestorAATion, Wave achieved its first WVE-006 milestone under the
collaboration, resulting in a $20 million payment from GSK. Wave is
eligible to receive up to $505 million in additional development,
launch, and commercial milestone payments, as well as tiered
royalties on net sales, from GSK for WVE-006. Development and
commercialization responsibilities will transfer to GSK after Wave
completes the RestorAATion-2 study.
- Beyond WVE-006, Wave is advancing a pipeline of wholly owned
RNA editing therapeutics across a range of high-impact
GalNAc-hepatic and extra-hepatic targets.
- Expected milestones: Wave plans to deliver
proof-of-mechanism data in individuals with AATD in 2024.
Advance INHBE program – first Wave wholly owned program
to emerge from GSK collaboration – as well as progress GSK
discovery programs under strategic collaboration
INHBE Program in Obesity
- Wave is advancing a novel approach for treating obesity with
its GalNAc-small interfering RNA (siRNA) targeting INHBE, which is
designed to induce fat loss while preserving muscle mass to restore
and maintain metabolic health.
- This program is designed to silence the INHBE gene through RNA
knockdown, thereby recapitulating the cardiometabolic profile of
INHBE loss of function (LoF) heterozygous human carriers. These
individuals exhibit reduced waist-to-hip ratio and reduced odds of
type 2 diabetes and coronary artery disease. INHBE reduction of 50%
or greater is expected to restore and maintain a healthy metabolic
profile, while preserving muscle mass.
- In September 2023, Wave demonstrated the first in vivo
proof-of-concept for INHBE silencing, with INHBE mRNA reduction in
diet-induced obese mice well beyond the 50% therapeutic threshold,
leading to substantially lower body weight and reduction of
visceral fat as compared to control.
- Expected milestones: Wave has identified
potent and highly specific INHBE siRNA leads and now expects to
select an INHBE clinical candidate in the third quarter of 2024,
ahead of expectation. This would enable clinical trial application
(CTA) submission in 2025.
GSK Discovery Collaboration
- Wave continues to advance its discovery collaboration with GSK,
which enables GSK to advance up to eight programs and Wave to
advance up to three programs (inclusive of INHBE) leveraging Wave’s
multimodal RNA medicines platform and GSK’s expertise in genetics
and genomics.
- Wave is eligible for up to $3.3 billion in potential milestone
payments for GSK’s eight collaboration programs and WVE-006, as
well as royalties on net sales.
- Expected milestones: Advance collaboration
activities with GSK, with potential for additional cash inflows in
2024.
Advance wholly owned DMD program with best-in-class exon
skipping to potentially registrational dystrophin data
WVE-N531 in Duchenne Muscular Dystrophy (DMD)
- Wave’s WVE-N531 program for boys with DMD amenable to exon 53
skipping is designed to induce production of endogenous, functional
dystrophin protein.
- Previous clinical data from the Phase 1b/2a Part A
proof-of-concept trial of WVE-N531 in DMD demonstrated high muscle
concentrations of WVE-N531 (mean of 42 micrograms/gram) and the
highest level of exon skipping ever seen in the clinic (mean exon
skipping of 53%; range: 48-62%) after three doses of 10 mg/kg every
other week. WVE-N531 appeared safe and well-tolerated, with all
treatment-related adverse events being mild.
- In December 2023, Wave announced the initiation of dosing in
the potentially registrational Phase 2 FORWARD-53 study and also
that the study was fully enrolled.
- Expected milestones: Wave expects to deliver
data, including dystrophin protein expression from muscle biopsies
at 24 weeks, in the third quarter of 2024.
Advance first-in-class, wild-type huntingtin-sparing HD
program and deliver multi-dose data set for
decision-making
WVE-003 in Huntington’s Disease (HD)
- WVE-003 is the most advanced investigational allele-selective
HD therapeutic designed to reduce mutant huntingtin (mHTT) protein
while also sparing healthy, wild-type huntingtin (wtHTT) protein,
and is currently being evaluated in the 30 mg every-eight-week
(Q8W) multi-dose cohort of the Phase 1b/2a SELECT-HD clinical
trial.
- In a non-human primate (NHP) study, WVE-003 achieved
significant tissue exposure levels in deep brain regions, including
the striatum. These data bolstered Wave’s existing datasets that
confirm the ability of its oligonucleotides to distribute to areas
of the central nervous system associated with HD.
- WVE-003 has demonstrated single-dose reductions in mean
cerebrospinal fluid mHTT of 35% compared to placebo, with
preservation of wtHTT, as previously shared in September 2022.
- Expected milestones: Wave expects to report
data from the 30 mg multi-dose cohort with extended follow-up,
along with all single-dose data, in the second quarter of 2024.
These data are expected to enable decision-making on the program
and support the opt-in package for Takeda.
Cash runway
- Wave expects that its cash and cash equivalents, together with
$115 million from its December 2023 offering and $27 million in
recent collaboration milestones in the fourth quarter of 2023, will
be sufficient to fund operations into the fourth quarter of 2025.
Wave does not include future milestones or other contingent
payments in its cash runway.
Upcoming events
- Paul Bolno, MD, MBA, President and Chief Executive Officer, is
scheduled to present at the 42nd Annual J.P. Morgan Healthcare
Conference in San Francisco, CA on Wednesday, January 10, 2024, at
3:00 p.m. PT / 6:00 p.m. ET. A live webcast of this presentation
will be available on the Investor Relations page of the Wave Life
Sciences website at http://ir.wavelifesciences.com. A replay of
this presentation will be archived and available on the website for
a limited time following the event.
About Wave Life SciencesWave Life Sciences
(Nasdaq: WVE) is a biotechnology company focused on unlocking the
broad potential of RNA medicines to transform human health. Wave’s
RNA medicines platform, PRISMTM, combines multiple modalities,
chemistry innovation and deep insights in human genetics to deliver
scientific breakthroughs that treat both rare and prevalent
disorders. Its toolkit of RNA-targeting modalities includes
editing, splicing, RNA interference and antisense silencing,
providing Wave with unmatched capabilities for designing and
sustainably delivering candidates that optimally address disease
biology. Wave’s diversified pipeline includes clinical programs in
Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and
Huntington’s disease, as well as a preclinical program in obesity.
Driven by the calling to “Reimagine Possible”, Wave is leading the
charge toward a world in which human potential is no longer
hindered by the burden of disease. Wave is headquartered in
Cambridge, MA. For more information on Wave’s science, pipeline and
people, please visit www.wavelifesciences.com and follow Wave on X
(formerly Twitter) and LinkedIn.
Forward-Looking Statements This press release
contains forward-looking statements concerning our goals, beliefs,
expectations, strategies, objectives and plans, and other
statements that are not necessarily based on historical facts,
including statements regarding the following, among others: the
anticipated initiation, site activation, patient recruitment,
patient enrollment, dosing, generation of data and completion of
our clinical trials, including any potential registration based on
these data, and the announcement of such events; our expectations
for 2024; our expectations to deliver the first-ever clinical
proof-of-mechanism data for RNA editing, with WVE-006 in AATD, and
our expectations for additional, wholly owned RNA editing programs;
the protocol, design and endpoints of our clinical trials; the
future performance and results of our programs in clinical trials;
future preclinical activities and programs; regulatory submissions;
the progress and potential benefits of our collaborations; the
potential achievement of milestones under our collaborations and
receipt of cash payments therefor; the potential of our preclinical
data to predict the behavior of our compounds in humans; our
identification and expected timing of future product candidates and
their therapeutic potential; the anticipated benefits of our
therapeutic candidates compared to others; our ability to design
compounds using multiple modalities and the anticipated benefits of
that approach; the breadth and versatility of PRISMTM; the expected
benefits of our stereopure oligonucleotides compared with
stereorandom oligonucleotides; the potential benefits of our RNA
editing capability, including our AIMers compared to others; the
potential benefits of our GalNAc-conjugated siRNA program targeting
INHBE; the status and progress of our programs relative to
potential competitors; the benefit of nucleic acid therapeutics
generally; the anticipated duration of our cash runway; our
intended uses of capital; and our expectations regarding any
potential global macro events beyond our control on our business.
Actual results may differ materially from those indicated by these
forward-looking statements as a result of various important
factors, including the following: our ability to finance our drug
discovery and development efforts and to raise additional capital
when needed; the ability of our preclinical programs to produce
data sufficient to support our clinical trial applications and the
timing thereof; the clinical results of our programs and the timing
thereof, which may not support further development of our product
candidates; actions of regulatory authorities and their
receptiveness to our adaptive trial designs, which may affect the
initiation, timing and progress of clinical trials; our
effectiveness in managing regulatory interactions and future
clinical trials; the effectiveness of PRISM; the effectiveness of
our RNA editing capability and our AIMers; our ability to
demonstrate the therapeutic benefits of our candidates in clinical
trials, including our ability to develop candidates across multiple
therapeutic modalities; our dependence on third parties, including
contract research organizations, contract manufacturing
organizations, collaborators and partners; our ability to
manufacture or contract with third parties to manufacture drug
material to support our programs and growth; our ability to obtain,
maintain and protect our intellectual property; our ability to
enforce our patents against infringers and defend our patent
portfolio against challenges from third parties; competition from
others developing therapies for the indications we are pursuing;
our ability to maintain the company infrastructure and personnel
needed to achieve our goals; and the information under the caption
“Risk Factors” contained in our most recent Annual Report on Form
10-K filed with the Securities and Exchange Commission (SEC) and in
other filings we make with the SEC from time to time. We undertake
no obligation to update the information contained in this press
release to reflect subsequently occurring events or
circumstances.
Investor Contact:Kate Rausch+1
617-949-4827krausch@wavelifesci.com
Media Contact:Alicia Suter+1
617-949-4817asuter@wavelifesci.com
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